Recruiting PHASE1 Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Tests treatment safety and results for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Official title Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003

ClinicalTrials.gov ID: NCT06714006

What this study is testing

What is PYC-003?

PYC-003 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for autosomal dominant polycystic kidney disease (adpkd).

Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.

What it's testing
This is a Phase 1, First-in-Human study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated Autosomal Dominant Polycystic Kidney Disease (ADPKD) There are 4 parts in this study, i.e. Part A, Part B, Part C and Part D.
  • Phase 1: an early, usually small safety study
  • Time commitment: about 96 weeks
  • You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.

A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.

Who can take part

Ages 18 to 65. Healthy volunteers may be eligible.

You may be able to join if

  • Part A (SAD - Healthy Volunteers) Key 1. Adult aged 18 to 60 years (inclusive)
  • At the discretion of the PI or designee, in good general health, with no significant medical history, and have no clinically significant...
  • BMI ≥ 18.0 and ≤ 32.0 kg/m2 and weight ≥ 50 kg.
  • Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.
  • Clinical laboratory values within normal range or deemed not clinically significant by the PI

You likely can't join if

  • Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.
  • Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with...
  • Has only 1 kidney or has received a kidney transplant.
  • Blood donation or had significant blood loss (\> 500 mL) within 30 days prior to the first administration of IP.
  • Plasma donation within 7 days prior to the first administration of IP.
  • Fever (body temperature \> 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP.
See the full eligibility criteria
Who can join
  • Part A (SAD - Healthy Volunteers) Key 1. Adult aged 18 to 60 years (inclusive)
  • At the discretion of the PI or designee, in good general health, with no significant medical history, and have no clinically significant abnormalities on physical examination at Screening
  • BMI ≥ 18.0 and ≤ 32.0 kg/m2 and weight ≥ 50 kg.
  • Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.
  • Clinical laboratory values within normal range or deemed not clinically significant by the PI
  • eGFR ≥ 80 mL/min/1.73 m2 via the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) 2021 calculation
  • Woman of childbearing potential (WOBCP) must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion.
  • Males must be surgically sterile (vasectomized for at least 6 months prior to first IP administration) or, if engaged in sexual relations with a WOCBP, must agree to use an acceptable, highly effective, double barrier...
  • Females must agree not to donate ova from the first administration of IP until 30 days following study completion.
  • Males must agree not donate sperm from the first administration of IP until 90 days following study completion.
  • Able and willing to attend the necessary visits to the study site.
  • Able and willing to adhere to caffeine, alcohol, and nicotine-containing product restrictions
  • Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures. Part A (SAD - Healthy Volunteers) Key
What rules you out
  • Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.
  • Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per protocol.
  • Has only 1 kidney or has received a kidney transplant.
  • Blood donation or had significant blood loss (\> 500 mL) within 30 days prior to the first administration of IP.
  • Plasma donation within 7 days prior to the first administration of IP.
  • Fever (body temperature \> 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP.
  • Infections requiring parenteral antibiotics within 6 months prior to Screening.
  • Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), HIV antibody.
  • History of life-threatening infection (e.g., meningitis).
  • Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.
  • Poor venous access.
  • History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.
  • History of malignancy, except for non-melanoma skin cancer excised more than 1 year prior to Screening and cervical intraepithelial neoplasia that has been successfully cured more than 2 years prior to Screening.
  • Abnormal electrocardiogram (ECG) findings at Screening, Day -3 to Day -1, or predose that are considered by the PI or designee to be clinically significant.
  • History or presence of a condition associated with significant immunosuppression.
  • Exposure to any drugs that cause significant immunosuppression (including experimental therapies as part of a clinical study) within 4 months or 5 half-lives (whichever is longer), prior to Screening.
  • ALP, AST, and ALT \> 1.5 × ULN at Screening or Day -3 to Day -1.
  • History of borderline to low blood magnesium and potassium levels and/or Screening or Day -3 to Day -1 blood magnesium level \< 0.7 mmol/L and potassium levels \< 3.5 mmol/L.
  • Active infection of the urinary tract (ie, kidney, bladder).
  • Positive toxicology screening panel (urine test including qualitative identification of amphetamines, barbiturates, benzodiazepines, cocaine, methamphetamine, methadone, opiates, phencyclidine, tetrahydrocannabinol...
  • History of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).
  • Regular alcohol consumption defined as \> 14 standard drinks per week for females and \> 21 standard drinks for males (where 1 standard drink = 375 mL of mid-strength beer [3.5% alcohol/volume], 100 mL wine [13.5%...
  • Unwilling to abstain from alcohol for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  • Use of any investigational medical device or investigational drug within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first administration of IP.
  • Use of (or anticipated use of) the following:
  • Any prescription drugs (other than hormonal contraception; oral contraceptive pills, long-acting implantable hormones, injectable hormones, a vaginal ring, or an intrauterine device [IUD]) within 14 days prior to the...
  • Any over-the-counter medication, herbal remedies, supplements or vitamins within 7 days prior to the first administration of IP and during the course of the study without prior approval of the PI and MM. Note...
  • Unwilling to refrain from strenuous exercise (including weightlifting) for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  • Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data. Part B and Part C (ADPKD Participants) Key...
  • Male or female aged 18 to 65 years (inclusive) at the time of informed consent.
  • ADPKD diagnosis as confirmed by the presence of genetic mutations associated with ADPKD, including, but not limited to, the presence of PKD1 mutation. Note: Where genotyping is not included the medical history for a...
  • Class 1C, 1D, or 1E per Mayo Imaging Classification System for Predicting Kidney Outcomes in ADPKD (Irazabal et al. 2015) (based upon prior magnetic resonance imaging [MRI] or computed tomography [CT] scan obtained...
  • BMI ≥ 18.0 and ≤ 35.0 kg/m2 and weight ≥ 50 kg.
  • Non-smoker and must not have used any tobacco or nicotine products within 2 months prior to Screening.
  • Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2 via the CKD EPI 2021 calculation 8\. Hematology and serum chemistry results at Screening that meet the following criteria:
  • Platelets \> 150 × 10\^9/L
  • Total white blood cell count \> 3.0 × 10\^9/L
  • Absolute neutrophil count \> 1.5 × 10\^9/L
  • Hemoglobin \> 110 g/L for females and \> 120 g/L for males
  • Total and direct bilirubin \< 1.5 × ULN, unless elevated bilirubin is associated with a known benign condition (e.g., Gilbert's syndrome)
  • Alanine aminotransferase (ALT) \< 1.5 × ULN
  • Aspartate aminotransferase (AST) \< 1.5 × ULN
  • Alkaline phosphatase (ALP) \< 1.5 × ULN
  • Gamma-glutamyl transferase \< 2 × ULN 9.WOCBP must agree to use an acceptable, highly effective, double barrier method of contraception from the start of Screening until study completion 10\. Males must be surgically...
  • Females who are pregnant, breastfeeding, or plan to become pregnant during the course of the study.
  • Presence of potentially confounding genetic mutations (per genotyping by a NATA accredited or equivalent diagnostic laboratory)including, but not limited to, the presence of PKD2, HNF1B, GANAB, IFT140, and/or DNAJB 11...
  • Use of (or anticipated use of) Tolvaptan and/or metformin administration within 30 days prior to the first administration of IP until study completion.
  • Underlying physical or psychological medical condition that, in the opinion of the PI or designee, would make the participant unlikely to comply with the protocol or complete the study per protocol.
  • Any renal or systemic pathology other than ADPKD or any other condition or prior therapy that in the opinion of the PI or designee would make the participant unsuitable for this study.
  • Has only 1 kidney or has a kidney transplant.
  • Blood donation or had significant blood loss (\> 500 mL) within 30 days prior to the first administration of IP.
  • Plasma donation within 7 days prior to the first administration of IP.
  • Has received (or is anticipated to receive) cell therapy, gene therapy, or RNA therapy for any renal condition.
  • Fever (body temperature \> 38°C) or symptomatic viral or bacterial infection within 2 weeks prior to first administration of IP.
  • Infections requiring parenteral antibiotics within 6 months prior to Screening.
  • Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), HIV antibody.
  • History of life-threatening infection (e.g., meningitis).
  • Vaccination with a live vaccine within 4 weeks prior to the first administration of IP.
  • Poor venous access.
  • History of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.
  • History of malignancy, except for non-melanoma skin cancer excised more than 1 year prior to Screening and cervical intraepithelial neoplasia that has been successfully cured more than 2 years prior to Screening.
  • Abnormal ECG findings at Screening, Day -3 to Day -1, or predose that are considered by the PI or designee to be clinically significant.
  • Abnormal vital signs findings at Screening that are considered by the PI or designee to be clinically significant. Note: A hypertensive participant is eligible if on a stable antihypertensive regimen for ≥ 28 days prior...
  • History or presence of a condition associated with significant immunosuppression.
  • Exposure to any drugs that cause significant immunosuppression (including experimental therapies as part of a clinical study) within 4 months or 5 half-lives (whichever is longer), prior to Screening.
  • History of borderline to low blood magnesium and potassium levels and/or Screening or Day -3 to Day -1 blood magnesium level \< 0.7 mmol/L and potassium levels \< 3.5 mmol/L.
  • Urine protein: creatinine ratio (UPCR) of \> 50 mg/mmol.
  • Hematuria (urine protein: creatinine ratio \> 30 mg/mmoL, or hematuria \> ++ on dipstick, or \> 100 cells per high-power field on microscopy) and/or urinary abnormalities at Screening deemed by the PI or designee to be...
  • Renal complications (eg, cyst rupture or cyst infections) within 6 weeks prior to first administration of IP.
  • Active infection of the urinary tract (ie, kidney, bladder).
  • Positive toxicology screening panel (urine test including qualitative identification of amphetamines, barbiturates, benzodiazepines, cocaine, methamphetamine, methadone, opiates, phencyclidine, tetrahydrocannabinol...
  • History of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).
  • Regular alcohol consumption defined as \> 14 standard drinks per week for females and \> 21 standard drinks for males (where 1 standard drink = 375 mL of mid-strength beer [3.5% alcohol/volume], 100 mL wine [13.5%...
  • Unwilling to abstain from alcohol for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  • Use of any investigational medical device or investigational drug within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first administration of IP.
  • Unwilling to refrain from strenuous exercise (including weightlifting) for 48 hours prior to admission to the study site and for 48 hours prior to any follow-up visits.
  • Anything that the PI considers would jeopardize the safety of the participant, prevent complete participation in the study, or compromise interpretation of study data. Part D will be an extension study for participants...

The study team makes the final eligibility decision.

Where it's taking place

  • Southport, Gold Coast, Australia
  • Concord, New South Wales, Australia
  • Liverpool, New South Wales, Australia
  • Wahroonga, New South Wales, Australia
  • Westmead, New South Wales, Australia
  • South Brisbane, Queensland, Australia
  • Fitzroy, Victoria, Australia
  • Saint Albans, Victoria, Australia
  • Joondalup, Western Australia, Australia
  • Takapuna, Auckland, New Zealand

Compensation & support

Compensation mentioned.

ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.

Questions & answers

Do participants get paid in this trial?

This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The study runs about 96 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.

Who can join this trial?

This study is enrolling all sexes, 18 years to 65 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Southport, Gold Coast, Australia; Concord, New South Wales, Australia; Liverpool, New South Wales, Australia; Wahroonga, New South Wales, Australia; Westmead, New South Wales, Australia; South Brisbane, Queensland, Australia and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.